Roche’s SMA Candidate Risdiplam Gets PRIME Designation

spinal muscular atrophy

Roche as announced the EMA has granted PRIME (Priority Medicines) designation to the company’s investigational oral medicine risdiplam (RG7916) for the treatment of people with spinal muscular atrophy (SMA).  The EMA grants PRIME status to drugs that have a major therapeutic advantage over existing treatments, or benefits patients without treatment options.  Developed by Roche, PTC Therapeutics and SMA Foundation to treat SMA, it is a pyridazine derivative that works by increasing the amount of functional survival of motor neuron protein product by the SM2 gene through a modifying its splicing pattern.